Gain Therapeutics' Gaucher, Parkinson's Candidate Shows Potential In Patient-Derived iPSC Study

The topline data from a study conducted at the University of Maryland School of Medicine (UMSOM) by Gain Therapeutics Inc GANX evaluating two lead Structurally Targeted Allosteric Regulators (STARs) compounds, GT-02287 and GT-02329, for Gaucher and GBA1 Parkinson's Disease, demonstrated positive effects on all tested phenotypes.
The study used human induced pluripotent stem cells (iPSC) derived from GD and GBA-associated Parkinson's disease patients.
Data showed that the compounds increased the levels of GCase protein, its transport to the lysosome, and its enzymatic activity.
In dopaminergic neurons, the two lead STAR chaperones also decrease the levels of α-synuclein-p129, demonstrating their potential to treat GBA1-associated Parkinson's Disease.
The Company anticipates initiating IND-enabling studies for Gaucher / Parkinson's Disease in Q4 of 2021.
Price Action: GANX stock is up 11.14% at $8.78 during the market session on the last check Wednesday.